Long-term safety and effectiveness study of vatiquinone (oral capsules) in patients with Friedreich ataxia who have previously received this treatment

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What is this study about?

This study focuses on Friedreich ataxia, a rare genetic disorder that affects the nervous system and causes progressive damage to the spinal cord and nerves. The study will evaluate a medication called vatiquinone (also known as PTC743), which is being developed to treat conditions affecting cellular energy production.

The purpose of this research is to assess the long-term safety of vatiquinone in people with Friedreich ataxia who have previously received this medication in other studies. The medication comes in the form of capsules that are taken by mouth. Participants will receive vatiquinone for up to 36 months, with a maximum daily dose of 200 mg.

During the study, participants will be monitored for any health changes and will undergo various medical assessments. These assessments will include checking their ability to maintain balance while standing, movement of arms and legs, and speech function. The study will track how these abilities may change over time while taking the medication.

1 Initial medication administration

You will receive vatiquinone in the form of oral capsules

This medication is intended for patients with Friedreich ataxia who have previously taken vatiquinone in another clinical study

2 Contraception requirements

If you are able to have children, you must use effective contraception methods from the start of the study until 30 days after the last dose

Acceptable methods include: implants, injections, patches, oral contraceptives, barrier methods, or intrauterine devices

Male participants must not donate sperm during the study and for 30 days after the last dose

If no other contraception method is used, a double-barrier method (condom with spermicide) is required

3 Safety monitoring

Your health will be monitored through regular checks of:

– Adverse events and serious adverse events

– Laboratory tests

– Vital signs

– Heart recordings (ECGs)

4 Assessment of condition

Your condition will be evaluated using the mFARS scale, which measures:

– Ability to maintain upright position

– Upper arm and hand function

– Lower limb function

– Speech and swallowing function

5 Study duration

The study continues until December 31, 2027

Your participation may end earlier if needed

Who Can Join the Study?

Must have previously participated in and completed a clinical study using vatiquinone (an investigational medication)
Must be willing to provide informed consent to participate in the study
If you are able to have children (both men and women), you must agree to use effective birth control methods such as:
- Implants
- Injectable contraceptives
- Birth control patch
- Birth control pills
- Barrier methods (like condoms)
- Intrauterine devices (IUD)
Must use birth control from the time of signing consent until 30 days after the last dose of study medication
Male participants must agree not to donate sperm during the study and for 30 days after the last dose
If no other birth control method is being used, participants must use a double-barrier method (condom with spermicide)
Both men and women can participate in this study
The study includes adults and adolescents

Who Cannot Join the Study?

Having any medical condition that could interfere with the safe conduct of the study, as determined by the study doctor
History of significant allergic reactions or hypersensitivity to vatiquinone or similar medications
Pregnancy or breastfeeding
Participation in another clinical trial within the past 30 days
Inability to comply with study procedures and follow-up visits
Having any unstable medical conditions that require immediate treatment
Use of prohibited medications that might interact with the study drug
Significant abnormalities in blood tests or other laboratory results that could affect safety
History of substance abuse within the past year
Mental conditions that could affect the ability to provide informed consent or follow study instructions

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name	City	Country
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Universitaetsklinikum Tuebingen AöR	Tuebingen	Germany
Hopital Beaujon	Clichy	France
Ospedale Pediatrico Bambino Gesu’	Rome	Italy

Trial status

Country	Status	Recruitment Start
France	Recruiting	08.12.2022
Germany	Recruiting	08.12.2022
Italy	Recruiting	08.12.2022
Spain	Recruiting	08.12.2022

Trial locations

Investigated drugs:

Vatiquinone

Vatiquinone is a medication being studied for treating Friedreich Ataxia, a rare genetic disease that affects the nervous system and heart. This drug is designed to help protect cells from damage caused by the disease. It works by supporting the function of mitochondria (the energy-producing parts of cells) and may help reduce oxidative stress, which is thought to play a role in the progression of Friedreich Ataxia.

Investigated diseases:

Friedreich's ataxia

Friedreich ataxia – A genetic neurological disorder that affects the nervous system and causes progressive damage to the spinal cord and nerves. The condition typically begins in childhood or adolescence and causes problems with movement and coordination. People with this condition experience progressive difficulty with balance, walking, and speech clarity. The disorder also commonly affects fine motor skills in the hands and arms. Friedreich ataxia can lead to changes in heart function and may cause curvature of the spine.

Trial ID:

2024-516505-23-00

Protocol code:

PTC743-NEU-003e-FA

NCT ID:

NCT05515536

Trial Phase:

Therapeutic confirmatory (Phase III)

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Long-term safety and effectiveness study of vatiquinone (oral capsules) in patients with Friedreich ataxia who have previously received this treatment

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?